Cycle 1 2018: Pragmatic Clinical Studies to Evaluate Patient-Centered Outcomes Town Hall January 29, 2018
Agenda • Welcome • Pragmatic Clinical Studies Submit questions via the chat function in Funding Announcement GoToWebinar. • Patient and Stakeholder Engagement Ask a question via phone at the end of the • Administrative Requirements presentation. • Merit Review • Resources • Questions?
Today’s Presenters Iris Giggetts, MSW, CRA Nora McGhee, PhD Denese Neu, PhD, MS David Hickam, MD, MPH Engagement Officer Senior Contract Administrator Senior Merit Review Officer Program Director Public and Patient Contracts Management and Office of the Chief Science Clinical Effectiveness and Engagement Administration Officer Decision Science
Pragmatic Clinical Studies Funding Announcement
Focus on Comparative Clinical Effectiveness Research (CER) CER includes: • Studies that compare the clinical effectiveness, benefits, and harms of two or more approaches to health care – Specific treatments and procedures – Complex interventions intended to improve healthcare delivery • All applicants should: – Explain how the research is comparative – Name the comparators – State why the comparisons are important
What is the Starting Point of Comparative Effectiveness? • Examine the choices people make about the options for managing a disease and improving healthcare outcomes • Consider how the need to compare these options could inform the focus of new research – Heterogeneity of the patient population – Understanding the important benefits and harms – Clarity about gaps in the current evidence base • Engagement with partners facilitates these steps
What is a Pragmatic CER Study? • Answers a practical, real world comparative effectiveness research question — a decisional dilemma • Assesses whether two or more options differ in effectiveness when administered as they are in real life (versus tightly controlled research situations) • The project is conducted in a clinical setting that is as close as possible to a real world setting • Has a sufficient sample size to provide insight on heterogeneity of treatment effects • The methodological approach (including study design, outcome measures, and follow up) is as simple as possible without sacrificing scientific rigor
Research We Do Not Fund PCORI does not fund research whose findings will include • cost-effectiveness analyses (CEA) • development of clinical practice guidelines • payment and coverage recommendations • or policy recommendations NOTE: PCORI does fund studies that explore the burden of costs on patients — for example, out-of-pocket costs.
PFA Overview: Pragmatic Clinical Studies to Evaluate Patient-Centered Outcomes Objective of this PFA: • Address critical clinical and health- Available Funds and Duration: related comparative effectiveness questions faced by patients, their • A total of $90 million caregivers, and their clinicians (direct and indirect) for this cycle In this PFA we seek to fund: • Up to $10 million in total • Large clinical trials that use efficient direct costs per project • Projects should be approaches completed within 5 years • Large scale observational studies
Comparators of Interest • Specific drugs, devices, and procedures • Techniques for behavioral modification • Complementary and alternative medicine • Delivery-system interventions
Potential for the Study’s Findings to be Adopted into Clinical Practice and Improve Delivery of Care • Describe how evidence generated from this study could be adopted into clinical practice and care delivery – Who are the end-users and how would the information from this study support a demand for information from end- users? – How likely are the findings to be reproduced by others? What are the barriers? – What is the dissemination plan for study results, beyond traditional publication and presentation? • Partnership with regional and/or national partner organizations
The Case of Usual Care • “Usual care” is typically a suboptimal comparator for CER studies. • It is ill-defined, difficult to quantify, and subject to considerable geographic and temporal variations, thus limiting interpretability, applicability, and reproducibility. • If the applicant proposes “usual care” as a rational and important comparator in the proposed study, then it must be described in detail, coherent as a clinical alternative, and properly justified as a legitimate comparator (e.g., usual care is guidelines-based). • The applicant needs to address why usual care is being proposed instead of an active comparator. • Additionally, it should be accompanied by an explanation of how the care given in the usual care group will be measured and how appropriate inferences will be drawn from its inclusion.
Justification for the Interventions that will be Studied (Including Complex Interventions) • Should refer to existing efficacy or effectiveness studies • Document prior evidence of efficacy or effectiveness of components of current interventions, or of effectiveness of the interventions in smaller studies or other settings • For studies conducted in patient populations that have experienced disparities, refer to existing efficacy or effectiveness studies in the target population where possible, or highlight efficacy or effectiveness studies in the general population that may be promising for the target population
PCORI Methodology Standards In any study, methods are critical. PCORI’s Methodology Committee developed Methodology Standards to which patient-centered CER must adhere The 48 standards can be grouped into 2 broad categories and 12 topic areas. Cross-Cutting Standards Design-Specific Standards • • Formulating Research Questions Data Registries • • Patient Centeredness Data Networks • • Data Integrity & Rigorous Analyses Causal Inference Methods* • • Preventing/Handling Missing Data Adaptive & Bayesian Trial Designs • • Heterogeneity of Treatment Effects Studies of Medical Tests • Systematic Reviews • Research Designs Using Clusters 14
PCS Priority Topics • PCORI’s multi -stakeholder panels have identified 16 high- priority topics and research questions – First consideration will be given to applications that directly address one or more of the 16 PCORI identified topics • Note that PCORI is open to receiving and reviewing LOIs for studies on investigator-initiated CER questions • AHRQ Future Research Needs Projects • IOM 100 priority topics for CER • Researchers must make a strong case for the importance of the proposed research. Describe clearly the evidence gap that the study will fill.
CEDS Priority Topics • Treatment of anxiety in children, adolescents, and young adults • Pharmacologic, psychological, or combination treatments for treating different types of insomnia • Alternative antibiotic regimens for empiric outpatient treatment of adults with community-acquired pneumonia • Treatments for intermediate- or high-risk non-invasive bladder cancer who have failed first-line intravesical therapy • Surgical treatments for acute hip fracture in elderly patients • Treatment strategies for symptomatic osteoarthritis (OA), including joint replacement
HDDR Priority Topics • Delivery modes for screening, Brief Intervention, and Referral to Treatment for adolescent alcohol abuse • Multicomponent interventions to reduce initiation of tobacco use and promote cessation of tobacco use among high-risk populations with known disparities • Integration of mental and behavioral health services into the primary care of persons at risk for disparities in health care and outcomes • Remote delivery approaches to non-pharmacological treatments for depression and anxiety conditions • Improving perinatal care in mothers and babies at risk for disparities • Reducing non-traumatic lower-extremity amputations in racial or ethnic minorities and low-income populations with diabetes • Comprehensive support services for infants and caregivers after discharge from the neonatal intensive care unit • Alternative delivery models versus the dentist’s office in preventing dental caries in children in medically underserved areas. • Models for integrating pharmacists into the care transitions team. • Screening and primary prevention approaches to minimize adolescent suicidality.
What does PCORI look for when reviewing LOIs? • Importance and relevance of the topics to PCORI priorities, as evidenced by critical gaps identified by clinical guideline developers and/or a recent relevant systematic review. • Clarity and credibility of applicants’ responses to the LOI questions such as well-described comparators, clear research methods (e.g., study design, sample size, effect size) • Programmatic fit and balance 18
Patient and Stakeholder Engagement
Patients and Other Stakeholders Patient/ Consumer Caregiver/ Family Purchaser Member of Patient Payer Clinician PCORI Community Patient/ Caregiver Industry Advocacy Org Hospital/ Policy Health Maker System Training Institution
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